A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)

Phase 2

Terminated

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: Eteplirsen

• Registration Number: NCT03985878
• Lead Sponsor: Sarepta Therapeutics, Inc.

Brief Summary

The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-06-11
• Study First Submitted QC: 2019-06-11
• Study First Posted: 2019-06-14
• Study Start: 2019-06-26
• Primary Completion: 2022-08-31
• Study Completion: 2022-08-31
• Status Verified: 2023-07
• Results First Submitted: 2023-07-25
• Results First Submitted QC: 2023-07-25
• Last Update Submitted: 2023-07-25
• Results First Posted: 2023-08-18
• Last Update Posted: 2023-08-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 15

Inclusion Criteria

• Participant successfully completes 96 weeks of treatment in Study 4658-102.

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Exclusion Criteria

• Participant has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the participant, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.

Other inclusion/exclusion criteria apply.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Eteplirsen	Eteplirsen	Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.

Primary Outcome Measures

Name	Time	Method
Number of Participants Experiencing Adverse Events (AEs)	Up to 162 weeks	A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Death Due to Adverse Events	Up to 162 weeks	A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)	Up to 162 weeks	AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.

Trial Locations

Locations (4)

Hopital Trousseau, Bâtiment lemariey; 🇫🇷 Paris, France

UZ-Gent; 🇧🇪 Gent, Belgium

Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile; 🇮🇹 Rome, Italy

UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre; 🇬🇧 London, England, United Kingdom